CAMBRIDGE, Mass.--(BUSINESS WIRE)--eGenesis, a biotechnology company focused on transforming
xenotransplantation into a lifesaving medical procedure, announced the
publication of a study in the journal Science
by eGenesis scientists and their collaborators demonstrating the
inactivation of PERV to prevent cross-species viral transmission and a
breakthrough in producing the first PERV-free pigs, an important
milestone for xenotransplantation.

Xenotransplantation, the use of animal organs for human transplant, is a
promising approach to alleviate the severe shortage of organs for human
transplantation but the risk of cross-species transmission of PERVs,
among other issues, has to date impeded its use in humans. eGenesis is
committed to harnessing CRISPR technology to deliver safe and effective
human transplantable cells, tissues and organs grown in pigs, thus
addressing a dire need for hundreds of thousands of patients worldwide.

“This is the first publication to report on PERV-free pig production,”
said Luhan Yang, Ph.D., co-founder and chief scientific officer at
eGenesis. “We generated a protocol to enable multiplex genome editing,
eradicated all PERV activity using CRISPR technology in cloneable
primary porcine fibroblasts and successfully produced PERV-free piglets.
This research represents an important advance in addressing safety
concerns about cross-species viral transmission. Our team will further
engineer the PERV-free pig strain to deliver safe and effective
xenotransplantation.”

This study examined the risk of PERV infectivity and demonstrated in
vitro that PERVs infected human cells and were transmitted to human
cells that had no history of contact with porcine cells in the
co-culture condition, substantiating the need to address this issue in
order to ensure safe xenotransplantation practice.

Researchers developed a strategy to enable efficient and precise genome
editing in primary cells using CRISPR-Cas9 technology. In conjunction
with a method to inhibit primary cell death during multiplex genome
editing, researchers successfully produced viable PERV-free porcine
embryos via somatic cell nuclear transfer, using engineered primary
cells. They then implanted the PERV-free embryos into surrogate sows and
demonstrated the absence of PERV re-infection, initially in fetuses and
finally in recently born piglets. These piglets are the first animals
born free of endogenous virus and will be monitored for any long-term
effects and impact by the eGenesis team.

The eGenesis team, having produced the first piglets free of active
PERVs, is working toward combining the safety benefits of PERV-free pigs
with additional gene editing addressing immunological response to
increase organ immune and functional compatibilities.

About CRISPR Gene Editing Technology

CRISPR is a genome-editing tool that can selectively delete, modify or
correct a disease causing abnormality in a specific DNA segment. CRISPR
refers to Clustered Regularly Interspaced Short Palindromic Repeats
occurring in the genome of certain bacteria. CRISPR technology uses a
protein-RNA complex composed of either the protein Cas-9 or Cpf1, each
of which binds to a guide RNA (gRNA) molecule that has been designed to
recognize a particular DNA sequence.

About Xenotransplantation

Currently in the United States, there is a tremendous unmet demand for
transplant organs with more than 118,000 people in need of a lifesaving
organ transplant. Of those, more than 75,000 people are active waiting
list candidates. The concept of cross-species transplantation, known as
xenotransplantation, is the transfer of living cells, tissues or organs
from one species to another. Due to the shortage of human organs,
xenotransplantation emerged as an alternative potential option and its
clinical potential is being explored with new technologies such as
CRISPR Cas-9. Xenotransplantation is not new – the first serious
attempts (then called heterotransplantation) first appeared in the
scientific literature in 1905 and it has been explored with limited
success over the last century.

About eGenesis

eGenesis is a biotechnology company focused on leveraging the
advancements of gene editing technologies to deliver safe and effective
human transplantable cells, tissues and organs to the hundreds of
thousands of patients worldwide who are in dire need.